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ShareScore release 0.9.0
Dataset results
13 results for “Oculopharyngeal muscular dystrophy”
Data from: Valproic acid is protective in cellular and worm models of oculopharyngeal muscular dystrophy
Objective: To explore valproic acid (VPA) as a potentially beneficial drug in cellular and worm models of oculopharyngeal muscular dystrophy (OPMD). Methods: Using a combination of live cell imaging and biochemical measures, we evaluated the potential protective effect of VPA in a stable C2C12 muscle cell model of OPMD, in lymphoblastoid cell lines derived from patients with OPMD and in a transgenic Caenorhabditis elegans OPMD model expressing human mutant PABPN1. Results: We demonstrated that VPA protects against the toxicity of mutant PABPN1. Of note, we found that VPA confers its long-term protective effects on C2C12 cell survival, proliferation, and differentiation by increasing the acetylated level of histones. Furthermore, VPA enhances the level of histone acetylation in lymphoblastoid cell lines derived from patients with OPMD. Moreover, treatment of nematodes with moderate concentrations of VPA significantly improved the motility of the PABPN-13 Alanines worms. Conclusions: Our results suggest that VPA helps to counteract OPMD-related phenotypes in the cellular and C elegans disease models.
Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts
ClinicalTrials.gov study NCT00773227. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Data from: Valproic acid is protective in cellular and worm models of oculopharyngeal muscular dystrophy
Open the record for dataset details and reuse information.
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
ClinicalTrials.gov study NCT02015481. IPD Sharing: Not stated. Countries: 3. Publications: 0.
Molecular and phenotypic characterization of a mouse model of oculopharyngeal muscular dystrophy reveals severe muscular atrophy restricted to fast glycolytic fibres
GEO Series GSE26604. Mus musculus. 33 samples. Type: Expression profiling by array.
Histopathology of the tongue in a mouse model of oculopharyngeal muscular dystrophy reveals progressive PABPN1 deposition associated with muscle wasting.
GEO Series GSE263433. Mus musculus. 8 samples. Type: Expression profiling by high throughput sequencing.
Dysphagia in Oculopharyngeal Muscular Dystrophy (OPMD)- Evaluation, Endoscopic Examination of Swallowing, Treatment and Long Term Follow up
ClinicalTrials.gov study NCT01167439. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft
ClinicalTrials.gov study NCT02878694. IPD Sharing: NO. Countries: 1. Publications: 0.
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
ClinicalTrials.gov study NCT06185673. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Screening in Oculopharyngeal Muscular Dystrophy
ClinicalTrials.gov study NCT02877784. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Effect of Aerobic Training in Patients With Oculopharyngeal Muscular Dystrophy
ClinicalTrials.gov study NCT02158156. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
ClinicalTrials.gov study NCT04226924. IPD Sharing: NO. Countries: 1. Publications: 0.
Natural History Study of Oculopharyngeal Muscular Dystrophy
ClinicalTrials.gov study NCT03161847. IPD Sharing: NO. Countries: 0. Publications: 0.
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Allen Brain Atlas
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DANDI Archive for NWB datasets
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International Brain Laboratory public data
The International Brain Laboratory public data releases expose standardized mouse decision-making experiments, including Neuropixels recordings, widefield calcium imaging, behavior, and session metadata accessed through the ONE API.
OpenNeuro
OpenNeuro is a free, open platform for sharing neuroimaging datasets, with public search, dataset pages, and download paths for web, S3, DataLad, and the OpenNeuro CLI.